Health Trials

Prion Disease

2

Review clinical trials related to Prion Disease. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

PrP-targeting siRNA Safety & Mechanism Study

The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.

Participants needed: 30
Trial details
Phase: Phase 1Age: 18+Biological sex: AllType: InterventionalSponsor: Broad Institute of MIT and HarvardUpdated: Jun 5, 2026Locations: 5
Eligibility criteria

clinically manifested symptoms of prion disease, in the opinion of the investiga... [+4]

pregnancy; [+2]

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Status: Recruiting

PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.

Participants needed: 76
Trial details
Phase: Phase 1, Phase 2Age: 18+Biological sex: AllType: InterventionalSponsor: Ionis Pharmaceuticals, Inc.Updated: Apr 24, 2026Locations: 13
Eligibility criteria

A confirmed diagnosis of probable or definite prion disease. [+4]

Clinically significant abnormalities in medical history, laboratory tests or phy... [+4]

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